AU2020371662A1 - Adeno-associated virus (AAV) systems for treatment of progranulin associated neurodegenerative diseases or disorders - Google Patents
Adeno-associated virus (AAV) systems for treatment of progranulin associated neurodegenerative diseases or disorders Download PDFInfo
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Classifications
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
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- A61P25/00—Drugs for disorders of the nervous system
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/475—Growth factors; Growth regulators
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- C07—ORGANIC CHEMISTRY
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16051—Methods of production or purification of viral material
- C12N2710/16052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2750/00011—Details
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- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
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- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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| US201962924340P | 2019-10-22 | 2019-10-22 | |
| US62/924,340 | 2019-10-22 | ||
| PCT/US2020/056860 WO2021081201A1 (en) | 2019-10-22 | 2020-10-22 | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders |
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| JP2021533126A (ja) * | 2018-07-31 | 2021-12-02 | コーネル・ユニバーシティーCornell University | 臓器機能を制御するための遺伝子治療方法 |
| KR20230128001A (ko) * | 2020-12-01 | 2023-09-01 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도 |
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| WO2024035649A1 (en) * | 2022-08-08 | 2024-02-15 | Shape Therapeutics Inc. | Compositions for progranulin expression and methods of use thereof |
| KR20250131772A (ko) * | 2022-11-13 | 2025-09-03 | 알렉시온 파마 인터내셔널 오퍼레이션즈 리미티드 | 전두측두엽 치매에 대한 유전자 요법 |
| WO2025251041A1 (en) * | 2024-05-31 | 2025-12-04 | Bluerock Therapeutics Lp | METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS |
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| WO2009089635A1 (en) * | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| JP5879256B2 (ja) * | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| WO2016125330A1 (ja) * | 2015-02-02 | 2016-08-11 | 原 英彰 | 網膜再生促進薬 |
| WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| WO2018136680A1 (en) * | 2017-01-19 | 2018-07-26 | New York University | Progranulin and progranulin derivatives in treating impaired fracture healing |
| EP3692075A4 (en) * | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| KR102709597B1 (ko) * | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| US20220111005A1 (en) * | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| BR112021015817A2 (pt) * | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
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| WO2021081201A1 (en) | 2021-04-29 |
| MX2022004812A (es) | 2023-02-23 |
| CA3158516A1 (en) | 2021-04-29 |
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| CN115715327A (zh) | 2023-02-24 |
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| US20240318199A1 (en) | 2024-09-26 |
| US20210147872A1 (en) | 2021-05-20 |
| JP2022553307A (ja) | 2022-12-22 |
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| EP4048799A1 (en) | 2022-08-31 |
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